Opus Genetics, Inc. has received FDA clearance for its investigational new drug (IND) application for OPGx-RDH12, a gene therapy for the treatment of RDH12-associated Leber congenital amaurosis (RDH12-LCA)
Have we heard about this therapy before?
This is actually the first time Glance is reporting on it …
Up until now, our coverage on Opus Genetics’ pipeline has extended to:
- OPGx-LCA5 for the treatment of LCA type 5, caused by biallelic mutations in the LCA5 gene
- Currently under phase 3 clinical evaluation
- See here for prior positive phase 1/2 pediatric data.
- Currently under phase 3 clinical evaluation
- OPGx-BEST1 for the treatment of bestrophine-1 (BEST1)-related IRDs
- Currently under phase 1/2 clinical evaluation
… and didn’t the company also just receive FDA approval for a third therapy?
Indeed it did—earlier this month, in fact.
Check out our coverage on the federal agency’s approval of RYZUMVI (phentolamine ophthalmic solution 0.75%) for the treatment of presbyopia in adults.
- For context: While Viatris, Inc. holds the exclusive U.S. commercialization rights of this prescription eye drop, it originated from Opus Genetics.
Gotcha. Alrighty, now let’s talk about OPGx-RDH12.
Before we do, it’s important to understand the inherited retinal disease (IRD) that the therapy is targeting: RDH12-LCA, a rare IRD that causes progressive vision loss and blindness beginning in early childhood.
- Mutations in the RDH12 gene impair protein function in the retina, leading to early visual decline.
- This is often accompanied by structural retinal changes (by age 2) and rapid progression during adolescence.
So how does OPGx-RDH12 address this?
The investigational adeno-associated virus (AAV)-based gene therapy is designed to deliver a functional copy of the RDH12 gene directly to photoreceptor cells in the retina.
And how has it performed in clinical evaluations?
Opus Genetics noted that preclinical studies of the therapy—conducted in cell and mouse models—supported further evaluations in humans.
- Specifically, the therapy demonstrated “restoration of RDH12 activity and functional improvements.”
Also worth noting: In 2022, the company reported promising results from two dose-ranging preclinical studies performed among nonhuman primates (NHP), in which subretinal delivery of the therapy (referred to as OPGx-002 at the time) was found to be safe.
Nice! What else?
The therapy is under development as part of Opus Genetics’ strategic collaboration with the Global DH12 Alliance comprising the U.S.-based RDH12 Fund for Sight and UK-based Eyes on the Future nonprofits.
Click here to read up on this partnership, which launched in July 2025 and includes the alliance committing up to $1.6 million toward OPGx-RDH12’s clinical development program.
How about regarding this IND?
Thanks to the FDA’s clearance, the company can now move forward with advancing OPGx-RDH12 into clinical development—for humans, this time.
Tell me more.
Per CEO George Magrath, MD, a phase 1/2 trial is expected to kick off before the end of the year (Q4 2026).
“Receiving IND clearance for OPGx-RDH12 represents an important step toward bringing a potentially transformative gene therapy to children and families affected by this devastating disease,” he stated.
Do we know any study details?
Unfortunately, no further information on the study (including participation, design, outcome measures, and expected data readout timeframe details) were shared.
So you know what that means … stay tuned!